UPDATED: Once stiff-armed at the FDA, new management offers Amicus a warm embrace for Galafold — priced at $315,000

by John Carroll — on August 10, 2018 04:40 PM EDTUpdated: August 13, 2018 08:49 AM

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It turns out the FDA wasn’t done Friday with the historic approval for Alnylam’s world’s-first RNAi drug. After the market close, Amicus Therapeutics $FOLD revealed that the agency had also come through with an OK for Galafold (migalastat), a controversial new med that had initially been firmly rejected by the agency.

On second glance, which came with an accelerated priority review and some red-carpet treatment at the agency, the FDA determined that Galafold had everything they were looking for in a drug for Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data.

Up to half of the 3,000 or so US patients with Fabry’s disease could qualify for this drug, which treats a rare enzyme deficiency. And they will be considering a drug that comes with a price of $315,000 a year, according to a Reuters report on Monday morning. That may look high, but it’s well within the usual norm for six-figure prices associated with therapies aimed at a rare disease. Amicus is also outlining plans to keep future price hikes in line with inflation.

The FDA’s initial position on Galafold was that it had no reason to be approved at this stage, looking for a new Phase III to justify a marketing OK. Now it came through with a green light several days ahead of the accelerated PDUFA date.

John Crowley

Amicus CEO John Crowley employed some dubious arguments as he fought vehemently against the rejection and a demand for a new Phase III study of gastrointestinal symptoms. Initially, the CEO had publicly told investors it would take 2 years to come up with the added data, which is about average for the task. But he privately told Janet Woodcock and others that the actual timeline would be 5 to 7 years, which made the development program impractical.

Crowley not only lobbied the newly elected President Donald Trump, he also implored the newly-selected FDA director Scott Gottlieb about the untenable demands in a letter. Gottlieb’s staff later said the chief never saw those arguments, sending it along to the right officials.

This was one of three drug programs that the FDA reversed itself on in the months after Gottlieb took over as commissioner at the FDA, with Eli Lilly and TherapeuticsMD — chaired by ex-HHS secretary Tommy Thompson — getting a do-over. The agency has never explained the rejections, never explained why it decided to reconsider their decision, or why it would offer Amicus a quick decision now. But all three won their approvals, though Lilly was left with a green light on a low dose that may have few takers.

There wasn’t a hint of any past objections in the FDA approval release Friday evening.

“Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease. Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research.

And you can be sure that at least a few regulators are praying this dramatic about-face won’t come back to haunt them later.

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